This study seeks to develop and validate non-invasive assessments of cardiac and respiratory muscles with magnetic resonance imaging (MRI) to better predict the natural disease progression of Duchenne muscular dystrophy (DMD) in affected individuals over time, as well as determine whether peripheral skeletal muscle dysfunction can predict cardiopulmonary dysfunction. The central hypothesis is that non-invasive MRI measures of the heart, muscle, and peripheral skeletal muscles can sensitively predict future cardiopulmonary decline.
Magnetic Resonance Imaging (MRI) of the heart and breathing muscles, special breathing tests (called pulmonary function testing), special exercise tests (using a stationary bike), and possibly an echocardiogram (ultrasound of the heart, commonly known as an "echo") will be completed up to 4 times per year for up to 4 years. Most participants will complete testing once or twice each year; however, some participants will be asked to repeat some of the tests twice during each visit.
Study Type
OBSERVATIONAL
Enrollment
9
It is a non-invasive method to determine ventricular size, volumes, mass, and ejection fraction.
It is non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
Metabolic exercise testing, including assessment of exercise capacity and MVO2, evaluates global cardiopulmonary functional status. This is performed with the use of a stationary bicycle.
Clinical and Translational Research Building
Gainesville, Florida, United States
Magnetic Resonance (MRI) T2 and Magnetic Resonance Spectroscopy (MRS)
The MRI T2 and MRS will be used as a noninvasive marker of myocardial damage/inflammation of participants of this study as an early detection for DMD.
Time frame: up to 4 years
Pulmonary Function Testing (PFT)
Non-invasive breathing tests that characterize respiratory muscle function, as well as lung compliance and physiology.
Time frame: up to 4 years
Metabolic Exercise Testing (exercise capacity and MVO2)
With the use of metabolic exercise testing, the aim is to correlate changes in cardiopulmonary function with decline in peripheral skeletal muscle function in individuals with DMD. Metabolic exercise testing includes measuring exercise capacity and maximum oxygen consumption (MVO2).
Time frame: up to 4 years
Multiple-echo Dixon
The echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure. This method and MRS will also be used for fat fraction determination.
Time frame: up to 4 years
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The echocardiogram performed with the multiple-echo Dixon method helps to assess participants cross-sectionally and longitudinally for variations and changes in myocardial structure.